Broad Institute

JUNE 10, 2024

The advance, from the lab of Broad core institute member David Liu , could one day help researchers develop a single gene therapy for diseases such as cystic fibrosis that are caused by one of hundreds or thousands of different mutations in a gene.

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DNA Engineering Therapies Disease 144

DrugBank

JUNE 13, 2024

Similarly, for cardiovascular diseases, implantable drug-eluting stents can deliver controlled doses of anti-proliferative drugs directly to the site of arterial blockage, preventing restenosis (re-narrowing) and improving long-term integrity of the vessel. 

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Chemical Biology and Drug Design

NOVEMBER 23, 2023

The symptoms of the disease include delusions and hallucinations, associated with extremely disordered behavior and thinking, which may affect the daily lives of the patients. Advancements in technology have led to understanding the dynamics of the disease and the identification of the underlying causes.

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Therapies DNA Regulations Disease 100

ASPET

SEPTEMBER 16, 2024

Glioblastoma (GBM) is a disease of the whole brain, with infiltrative tumor cells protected by an intact BBB. Standard of care GBM therapies include radiation and cytotoxic chemotherapy that lead to DNA damage. Subsequent activation of DNA damage response (DDR) pathways can induce resistance.

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Pharmacogenetics DNA Treatment Pharmacokinetics 100

DrugBank

JANUARY 9, 2025

Targeted Therapies vs. Traditional Treatments Traditional cancer treatments, such as chemotherapy and radiotherapy, are inherently cytotoxic, targeting both cancerous and healthy cells. This lack of selectivity often results in significant side effects, including nausea and limited efficacy, particularly in advanced or metastatic disease.

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Therapies Disease Treatment Small Molecule 52

Broad Institute

JANUARY 8, 2024

In the new work published today in Nature Biotechnology , the team adapted engineered virus-like particles (eVLPs) that they had previously designed to carry base editors — another type of precision gene editor that makes single-letter changes in DNA. By Sarah C.P.

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PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. million DNA bases.

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FDA Approval Treatment FDA DNA 96

Drug Target Review

JULY 7, 2023

The reasons for this are multifaceted, including concerns over the safety of directly altering DNA sequences and subsequent regulatory restrictions that have arisen as a result. The epigenome (meaning ‘above the genome’) is a system of reversible marks regulating how the DNA is read, translated and used. What is epigenetic editing?

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DNA Engineering Therapies Regulations 106

DrugBaron

JANUARY 8, 2021

Yesterday, Sarpeta (NASDAQ: $SRPT) announced that its gene therapy for Duchenne Muscular Dystrophy failed to improve muscle function in a study of 40 boys, despite achieving impressive expression of micro-dystrophin (at least in the short term). Surely, then, a correct set of DNA plans will ensure a correctly functioning cell?

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DNA Therapies Disease Production 100

Drug Target Review

SEPTEMBER 28, 2023

After some time in that role and launching several products, I received a call from Bill Banyai and Bill Peck, or ‘The Bills’ as we call them, who were building a company around technology that creates DNA by ‘writing’ it on a silicon chip. They needed reference materials for the disease in order to develop and validate diagnostic tests.

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DNA Organic Chemistry Science Virus 116

PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. DEB has been a candidate for a gene therapy since 2002.

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Drug Target Review

JANUARY 8, 2024

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

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Broad Institute

OCTOBER 23, 2024

They knew that discovering the genetic cause of her disorder would help them find other people like her, help get the condition formally recognized as a new disease, and help them better advocate for research into new treatments. This is the first human disease caused by loss of one copy of a lncRNA gene.

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DNA RNA Disease Hospitals 128

Broad Institute

JULY 10, 2024

We are hopeful that the use of prime editing to correct the predominant cause of cystic fibrosis might lead to a one-time, permanent treatment for this serious disease,” said Liu, the senior author on the study. There are more than 2,000 known variants of the CFTR gene, 700 of which cause disease.

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DrugBank

JANUARY 29, 2025

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Each of these mechanisms represents a distinct strategy to address the underlying genetic perturbations contributing to disease pathogenesis.

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Therapies DNA RNA Disease 52

KIF1A

APRIL 6, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. When a mutation causes a deletion or duplication of larger areas of DNA, we call it Copy Number Variation. She graduated from UC San Diego with a B.S.

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DNA Medical Schools Therapies Research 111

Broad Institute

NOVEMBER 13, 2023

Liquid biopsies enable clinicians to find and analyze tumor DNA in a patient’s blood sample to detect cancer early, monitor cancer recurrence, assess the patient’s response to treatment, and measure other clinically important features in real time, without invasive procedures.

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DNA Medical Schools Treatment Therapies 116

Broad Institute

OCTOBER 3, 2024

The findings point to factors involved in how neurons communicate and fire, suggesting potential targets for new therapies. Epilepsy also has several subtypes, and while one group called developmental encephalopathies have been connected to several genes, other forms of the disease are less well understood.

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DNA Doctors Treatment Disease 133

Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Hearing aids also have all sorts of deficits.

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Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

DrugBank

MARCH 12, 2025

However, mRNA technology is not limited to infectious diseases. This unique mechanism has enabled scientists to rethink how they tackle diseases, paving the way for advancements in oncology, genetic disorders, and regenerative medicine.  One of the most promising mRNA applications in this field is protein replacement therapy.

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Drug Target Review

AUGUST 3, 2023

Clinical trials are now underway for various genetic subtypes of both rare and common diseases, on the understanding that medications utilising genetic biomarkers have a significantly higher chance of success. There are clinical trials underway for genetic subtypes of rare and common diseases.

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DNA Clinical Trials Trials Disease 52

KIF1A

JANUARY 27, 2024

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. The study found improved spasticity and movement in mice treated with ARL61P1 gene therapy; it also restored abnormalities observed in the brains of ARL61P1-deficient mice.

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Therapies Small Molecule DNA Treatment 111

Drug Target Review

DECEMBER 5, 2024

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.

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Therapies Virus DNA Compliance 52

The Premier Consulting Blog

MARCH 7, 2024

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

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Production Therapies DNA Virus 52

Drug Target Review

NOVEMBER 6, 2023

Advances in disease understanding and combination strategies in haematology We now know that blood cancers are characterised by molecular and phenotypic heterogeneity. 2 It is this complexity that necessitates powerful, targeted combination therapies. 2 It is this complexity that necessitates powerful, targeted combination therapies.

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Small Molecule Therapies Treatment Disease 111

Drug Target Review

NOVEMBER 13, 2023

Regulatory T cells (Treg) cells are specialised immune cells that are essential for maintaining peripheral tolerance, preventing autoimmune diseases, and limiting chronic inflammatory diseases. Another finding was that regulatory T cells had distinct chromatin architecture features called DNA loops.

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Government DNA RNA Regulations 105

Drug Target Review

JUNE 9, 2023

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

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Treatment Packaging Clinical Research Therapies 106

DrugBank

JULY 24, 2024

This is a game-changer, especially in the fight against cancer and other complex diseases. For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed. This can potentially correct the root causes of some diseases at the genetic level.

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Small Molecule Drugs Clinical Trials Therapies 98

KIF1A

JANUARY 13, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. As one cell turns into two, its DNA must be replicated and then pulled apart, so that when the cell divides each has all the DNA it needs to function.

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DNA Regulations Drug Development Disease 105

NIH Director's Blog: Drug Discovery

MARCH 8, 2016

Instead of the black, printed stripes of the Universal Product Codes (UPCs) that we see on everything from package deliveries to clothing tags, they used short, unique snippets of DNA to label cells. DNA barcoding has already empowered single-cell analysis, including for nerve cells in the brain. PRISM consists of two key components.

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DNA Drugs Small Molecule FDA Approval 52

PLOS: DNA Science

FEBRUARY 8, 2024

Already, engineered probiotics have been used to treat metabolic disorders like inflammatory bowel disease and obesity, single-gene conditions like PKU, and bacterial infections common in people who have cystic fibrosis. Or, add genes to arm a host species to resist a specific infectious disease. appeared first on DNA Science.

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Immune Response DNA Engineering Virus 97

Drug Target Review

AUGUST 16, 2023

Furthermore, it successfully distinguished early-stage cancer samples from those with common diseases, laying the foundation for a robust data platform for further in-depth analysis. This in-depth understanding of the disease could aid in selecting targeted therapies for individual patients, further enhancing treatment outcomes.

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DNA Disease Therapies Treatment 98

Broad Institute

DECEMBER 13, 2023

Related news Researchers map brain cell changes in Alzheimer’s disease Courtesy of the Chen and Macosko labs. The researchers also discovered clues about cellular function and the potential roles of brain structures in disease. Courtesy of the Chen and Macosko labs. Our results underscore the need to study them more deeply.”

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The Pharma Data

DECEMBER 11, 2020

11, 2020 — Results from a long-term study of a gene therapy technique to prevent inherited mitochondrial disease show promise, researchers say. The researchers said the technique could break the cycle of disease passed from mother to baby through mutations in mitochondrial DNA (mtDNA). FRIDAY, Dec.

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Therapies Clinical Trials Disease Trials 52

DrugBank

MAY 15, 2024

Today, we're able to identify and target specific molecules involved in disease processes—a method that's much more like using a sniper rifle than throwing darts blindfolded. Among these targets are proteins, receptors, and enzymes that are fundamental to disease mechanisms.

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Small Molecule Molecular Biology Drugs Disease 98

PLOS: DNA Science

APRIL 18, 2024

Clues to combatting a devastating disease can come from identifying people who have gene variants – mutations – that protect them, by slowing the illness or lowering the risk that it develops in the first place. Understanding how they do this may inspire treatment strategies for the wider patient population. Quiroz, Ph.D.,

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Disease Research Therapies DNA 95

Codon

JUNE 11, 2024

Biologists use nanopores for everything from diagnosing diseases and monitoring changes within rainforest ecosystems to discovering proteins from microbes frozen in Icelandic glaciers. A scientist can isolate DNA and load up a flow cell in fifteen minutes. Nanopore devices work incredibly fast. An Oxford Nanopore device.

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